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ASH 2021 | Dec 11, 2021 - Dec 14, 2021

Rare Blood Disorders

Some of the most serious unmet patient needs today are in the field of hematology, and Sanofi Genzyme is committed to breaking barriers through groundbreaking science to significantly improve the health and lives of people with rare blood disorders around the world.

More about Rare Blood Disorders

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  • Hemophilia

    Hemophilia A and B are rare genetic blood disorders, characterized by excessive or uncontrolled bleeding into joints, tissues, and body cavities, which can be life-threatening or lead to disability.

  • aTTP

    Acquired thrombotic thrombocytopenic purpura (aTTP) is a rare blood disorder that develops when the immune system stops ADAMTS13, a protein (an enzyme), from working properly. Without this protein, the body creates too many blood clots.

  • CAD

    Cold Agglutinin Disease (CAD) is a rare type of chronic, autoimmune, hemolytic anemia. In people with CAD, the body’s immune system mistakenly attacks and destroys healthy red blood cells (hemolysis) by activating the "classical complement" pathway, which is part of the innate immune system. People with CAD may experience chronic anemia, profound fatigue, acute hemolytic crisis, and other potentially serious complications.

  • SCD

    Sickle cell disease is an inherited blood disorder that affects red blood cells. People with sickle cell disease have red blood cells that contain mostly hemoglobin S, an abnormal type of hemoglobin.

  • ITP

    Immune thrombocytopenia (ITP) is an autoimmune disorder characterized by the destruction of healthy platelets that increases a patient’s risk of bleeding​.